Company history
Vertex Pharmaceuticals (ticker: VRTX) was founded in 1989 by Joshua Berg and Kevin J. Kinsella.
Main products
The company mainly researches and produces various small molecule drugs for the treatment of terminal diseases. Its famous products include:
- Ivacaftor, marketed as Kalydeco, is used to treat cystic fibrosis. First approved in the United States in 2012, it is now available in more than 30 countries.
- Anti-hepatitis C drug Telaprevir.
- Casgevy therapy.
Drugs development in pipline
New triple combination cystic fibrosis therapy
Regulatory submissions in the U.S. and EU have been completed, and it primarily addresses 31 rare mutations that Vertex’s current line of drugs does not account for. More than 6,000 patients taking existing medications are estimated to be interested in this new treatment option.
Suzetrigine
Suzetrigine, another potential blockbuster product from Vertex, has begun Phase 3 trials as a non-opioid treatment for a variety of acute pain conditions. Vertex’s main target patients will be as a treatment for diabetic peripheral neuropathy (multiple acute pain ailments).
Gene editing therapy Casgevy
Vertex is launching Casgevy, a novel gene-editing therapy developed in partnership with CRISPR, as a one-time functional treatment for sickle cell disease and transfusion-dependent beta thalassemia.
This treatment is still in its early stages and is mainly targeted at some rare diseases; including:
- Alpha-1 antitrypsin (AAT) deficiency
- Beta thalassemia
- Sickle cell anemia
Major acquisitions
- On September 3, 2019, Vertex Pharmaceuticals spent US$950 million to acquire biotechnology company Semma Therapeutics.
- On April 11, 2024, Vertex Pharmaceuticals acquired the biotechnology company Alpine Immune for approximately US$4.9 billion in cash to obtain the latter’s drugs for the treatment of renal autoimmune diseases.
Main competitor
Bluebird Bio
The FDA approved Bluebird Biotechnology (ticker: BLUE)’s Lyfgenia for sickle cell disease in December, 2023, along with a rival gene therapy for the disease developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
It has gene-editing treatments for TDT and SCD on the market. But there are three reasons why it should not be a big threat:
- First, Bluebird Biotechnology only operates in the United States.
- Second, Vertex is a much larger biotech company than either Bluebird Biotech or CRISPR, and has a track record of negotiating lucrative deals with third-party payers.
- Third, Bluebird Biotech’s SCD treatment Lyfgenia carries a boxed warning for blood cancers. In the end, Lyfgenia cost $3.1 million, while Casgevy cost $2.2 million.
Capital market performance
Vertex Pharmaceuticals’ stock price rose by approximately 42.26% in one year in 2023; during the same period, CRISPR Therapeutics rose by 52.68%.
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